Article

Mavacamten improves energy balance in a pre-clinical model of RASopathy-associated hypertrophic cardiomyopathy

2025-02-27

Abstract excerpt

<h4>Background and aim</h4> Developmental disorders caused by activating mutations in the RAS-MAPK pathway account for nearly 20% of hypertrophic cardiomyopathy (HCM) cases in paediatric patients. Compared to sarcomeric HCM, RAS-HCM presents a higher risk of obstruction and hospitalisation. The myosin inhibitor mavacamten has been approved in the European Union for treating adults with obstructive HCM; however, c...

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Literature Corpus work: b04e3f2e-8146-5be1-af9c-36b176801335
DOI: 10.1101/2025.02.21.639222

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